NCT02432144

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of the study is to evaluate the long-term safety of UX003 in subjects with MPS 7.

Detailed description

Participants with MPS 7 who were UX003 treatment-naïve or previously enrolled and treated in a prior clinical study of UX003 (e.g. UX003-CL301 \[NCT02230566\], investigator sponsored trials, expanded access/compassionate use) can enroll into this treatment and extension study provided all eligibility criteria is met for a given participant.

Conditions

• Sly Syndrome
• MPS VII
• Mucopolysaccharidosis
• Mucopolysaccharidosis VII

Interventions

Type	Name	Description
DRUG	UX003	solution for intravenous (IV) infusion

Timeline

• Start date: 2015-11-10
• Primary completion: 2019-01-14
• Completion: 2019-01-14
• First posted: 2015-05-01
• Last updated: 2020-07-30
• Results posted: 2019-07-30

Locations

7 sites across 4 countries: United States, Brazil, Mexico, Portugal